Background and study aims: Crohn’s disease (CD) is often complicated by perianal fistulas or abscesses that worsen patient’s quality of life, with 52-88% of CD-related perianal fistulas (CPFs) being complex. This retrospective study describes sociodemographic characteristics, treatment patterns and outcomes of patients with complex CPFs in five European countries. Patients and methods: PREFACE is a multi-national medical chart review study of CD patients who started medical or surgical treatment for a new episode of complex CPFs between September 2011 and September 2014 (index date). Effectiveness outcomes were assessed as fistula remission rates based on fistula drainage assessment. The most recent assessments prior to each timepoint were used to calculate fistula remission rates. Results: In total, 372 patients (51.3% male, mean age at index date 37.8 years) with 498 complex CPFs between index date and end of data collection were included. A quarter of patients with CPF already had perianal fistulas at CD diagnosis. Of the complex index CPFs, 39.8% were treated with antitumour necrosis factor, 33.3% with antibiotics and 16.3% with immunosuppressants. At least one surgery was performed for 93.8% of complex CPFs. By end of follow-up (median (IQR) 6.0 (5.2, 6.9) years), 61.2% of complex CPFs were not draining anymore. Fistula remission rate at patient level was 28.0% after 6 months, 35.2% after 12 months and 64.8% overall. Conclusions: Current therapeutic algorithms for CPFs are not successful in a considerable proportion of patients. Improved therapeutic strategies and new treatment options are required to achieve better outcomes in complex CPFs.

PBM is a patient-centered approach that addresses iron deficiency, anaemia, coagulopathy, and blood loss, in surgical and nonsurgical patients, as risk factors for adverse medical outcomes. The objective is to assess the impact of PBM implementation on red blood cells (RBC) transfusion rate and length of hospital stay (LOHS).

PBM is a program including a set of evidence-based practices to optimize patient outcomes clinically managing and preserving the patient's own blood. The objective is to compare the transfusion rate and length of hospital stay (LOHS) before vs. after PBM implementation.

PBM aims to improve medical and surgical management of patients undergoing major elective surgery using techniques to manage and preserve the patient's own blood. The objective is to compare the length of hospital stay (LOHS) before vs. after the implementation of PBM in Humanitas Research Hospital of Milano, Italy.

Abstract Background Presence of fistulas in Crohn’s disease (CD) is an indicator of poor prognosis; 20% of CD patients suffer from perianal fistula. There are few studies specifically designed to assess treatment outcomes in complex perianal fistula (CPF) in CD. This retrospective chart review study describes the outcomes of patients with CPF in CD in five European countries after medical and/or surgical treatment. Methods Adult patients with CD receiving treatment for a new episode of CPF during the eligibility period (September 2011 to September 2014), in Belgium, France, Germany, Italy and Spain, were included. Index date was defined as date of any medical or surgical CPF treatment initiation. Data was collected from CD diagnosis to at least 3 years after index date (except for deceased or lost to follow-up patients) to describe patient characteristics and treatments used for all CPF episodes since CD diagnosis. Effectiveness outcomes were measured as remission rates based on Fistula Drainage Assessment (FDA) recorded in medical charts for fistula reported at index date (index fistula). Remission rates are expressed as percentage rates on patient level after 6- and 12-months follow-up period. For calculation of treatment outcomes, the most recent FDA prior to the respective timepoint was used. Results A total of 372 patients (51% male) with a mean (SD) age of 38 (13) were included by 31 sites. Median time since CD diagnosis was 7 years, and median length of follow-up was 6 years. A total of 498 CPFs were presented at index date and during FU period. Out of the 498 CPFs, 94% were treated with at least one surgical intervention (most frequent: 61% long-term seton placement, 51% surgical drainage) and 82% with at least one medical treatment (most frequent: 40% anti-TNFs, 33% antibiotics, 16% immunosuppressants). After 6 months the remission rate at patient level for index fistula was 28% and after 12 months 35%. Conclusion Current standards of care achieved remission in one third of patients with CPF in CD over a period of one year. Improved therapeutic strategies and new treatment options are required to improve outcomes in this manifestation of CD.

Abstract Background The α4β7 integrin monoclonal antibody vedolizumab (VDZ) has been shown to be efficacious for patients with moderate-to-severe Crohn’s disease (CD). This study aimed to analyse the added value of budesonide in combination with VDZ as an induction treatment for this indication. Methods A multicentre, retrospective chart review study was conducted in Belgium, Israel, and Switzerland. Adult patients with moderately to severely active CD (defined as an abdominal pain [AP] score of ≥2 and/or a mean daily loose stool frequency [LSF] score of ≥4 for the previous 7 days) who initiated induction therapy with either VDZ monotherapy (mono) or a combination therapy (combo) of VDZ with budesonide (index date) between 1 January 2015 and 31 January 2019 were included. Patients who received VDZ by IV infusion at weeks 0, 2, 6, 10 (only some patients received VDZ during week 10), and 8 weeks thereafter were assessed for time to patient-reported outcome (PRO) clinical remission (Kaplan-Meier curves), defined as an average daily composite score of AP ≤1 and LSF ≤31 within 14 weeks. Regression models were used to assess differences and associations. Results Overall, 123 patients were included (mono, n=73; combo, n=50). Patients initiating combo presented with more severe disease at index date than patients initiating mono. PRO clinical remission rates were estimated at 71.4% (50/70) in the mono and 68.0% (34/50) in the combo groups, with a similar median time to PRO remission of 91 days (95% CI: 70–98) and 95 days (95% CI: 70–98), respectively (Figure 1). Figure 2 shows the mean % change in AP and LSF from baseline to week 14, which was comparable for mono and combo. The variables associated with mean % change were moderate and severe AP scores for AP and being a current smoker for LSF. One patient in each group discontinued VDZ before week 14 (due to lack of effectiveness [mono] and adverse event [AE; combo]); 68.0% of patients in the combo group discontinued budesonide by the end of the follow up period. The reasons for discontinuation were routine treatment regimen (8 weeks 9 mg/day+subsequent tapering-off) in 85.3% of the patients, lack of effectiveness in 5.9% and AEs in 2.9% (5.8% other reasons). Safety event rates were similar among the groups for overall AEs (mono, 23.3%; combo, 26.0%), with the majority designated as mild to moderate in severity, and 83.3% resolved within the follow-up period. Conclusion Comparable effectiveness and safety outcomes were observed with mono and combo therapy in patients with CD; however, disease state among patients receiving combo was more refractory/severe at baseline. Further evidence is needed to corroborate these findings. Reference

There is limited information on healthcare resource utilization (HCRU) regarding complex perianal fistula (CPF) in Crohn’s disease (CD) in a multi-country setting. This study describes direct medical HCRU in patients with CD and CPF in five European countries. We conducted a retrospective medical chart review of patients with CD receiving treatment for a new episode of CPF from September 2011 to September 2014, in Belgium, France, Germany, Italy and Spain. Index date was defined as date of CPF-treatment initiation. Data on healthcare resources were collected for the follow-up period from index date for at least 3 years (except for deceased or lost to follow-up patients). In total, 372 patients (49% female) with mean (SD) age of 38 (13) years were included in the study by 19 gastroenterologists and 12 surgeons. Time since CD diagnosis was on average 10 (9) years, and length of follow-up 6 (1) years. For 95% of patients at least one visit to gastroenterologists and for 70% of patients at least one visit to a surgeon was documented in the follow-up period. One fifth had a requested visit (22%) or a visit due to systemic treatment administration (19%) beside scheduled follow-up visits. For most patients in Belgium (52%) and Spain (57%) an emergency room visit, and for most patients in Belgium (67%), Spain (57%) and Germany (54%) a hospitalization in wards other than ICU was reported. For more than 90% of patients liver function test, MRI, and total coloscopy/ileocoloscopy were performed with similar rates in most countries. Rates of emergency room visits and hospitalization are over 50% in some countries. There were no relevant differences regarding assessments or procedures between countries. MRI and total coloscopy/ileocoloscopy were performed in most patients. Further research is warranted to understand specific HCRU drivers in CPF in CD.

Abstract Background Presence of fistulas in Crohn’s disease (CD) is an indicator of poor prognosis; 22.1% of CD patients suffer from fistulising disease1 with high variability in complex perianal fistula (CPF) prevalence2. There is limited information available about the management of CPF in a real-world setting. This study describes the treatment patterns of patients with CPF in CD in Europe. Methods Retrospective medical chart review of consecutive patients with CD receiving treatment for a new episode of CPF during the period (September 2011 to September 2014), in Belgium, France, Germany, Italy and Spain. Index date was defined as the date of treatment initiation for a new episode of CPF during the eligibility period. Data was collected from CD diagnosis to at least 3 years after index date (except for deceased or lost to follow-up patients) to describe patient characteristics and treatments used for all CPFs episodes since CD diagnosis. Results A total of 386 patients (51% female) were included with a mean (SD) age of 38 (13) and 10 (9) years since CD diagnosis. At CD diagnosis, 28% of patients had ileal, 29% colonic and 39% ileocolonic involvement; 24% of study patients had anal or perianal fistula. Prior to index date, 42% of patients had at least one surgery, being partial resection of small bowel the most common one. ASA-5, anti-TNFs and immunosuppressants were used for CD or complications in 47%, 48% and 42% of patients. Patients presented 584 CPFs during the study period. More than half of these CPFs were trans-sphincteric (60%). Out of the 584 CPFs, 92% were treated with at least one surgical intervention (most frequent: 56% long-term seton placement, 46% surgical drainage), and 86.6% with at least one medical treatment. Medical treatments most frequently used for CPFs or CD and complications (overlapping a CPF episode) were anti-TNFs (49%), antibiotics (44%) and immunosuppressants (26%). Conclusion Almost one fourth of the patients with CPF already had anal or perianal fistulas at CD diagnosis. Based on ECCO guidelines it was expected that almost all CPFs in CD patients should be treated with anti-TNF with or without surgical intervention. However, the use of anti-TNF during CPF episodes was lower than expected. Surgical drainage and seton placement were performed in a majority of patients in at least 3 years following treatment intensification, with a low rate of other types of surgery. Almost two third of CPFs were trans-sphincteric and if inadequately treated, sphincter function may be compromised.

Evidence suggests that deficient mismatch repair (dMMR)/micrsatellite-High (MSI-H) metastatic colorectal cancer (mCRC) patients are less responsive to conventional treatment (chemotherapy +/- targeted therapy) and have a worse prognosis than those with MMR-proficient or microsatellite stable mCRC. Immune checkpoints inhibitors (ICIs) have rapidly emerged as a key treatment modality for dMMR/MSI-H tumors including mCRC. However, these data were based on single-arm clinical trials and clinical outcomes data with standard of care (SOC) regimens are lacking since dMMR/MSI-H advanced CRC represents only ∼5% of all CRC. The study objective was to evaluate real-world clinical outcomes in dMMR/MSI-H mCRC patients receiving third-line (3L) SOC regimens. Two tertiary French University hospitals participated in a retrospective chart review study in which adult patients previously diagnosed with mCRC (stage IV), with a dMMR and MSI-H status and treated with 2 or more prior lines of SOC therapy for advanced disease were enrolled. Key exclusion criterion was prior or current treatment for 3L with ICIs. A minimum of 6 months of follow-up was required, including patients who died during this time period. Overall survival (OS) from the start of 3L treatment (index date) was reported using Kaplan-Meier analysis. The best overall response rate (BORR) was reported along with complete response (CR) and partial response (PR). 36 dMMR/MSI-H mCRC patients were included. Majority were male (56%) with a mean age of 61.8 years and with synchronous metastatic disease (78%). RAS and BRAF mutations were observed in 31% and 42% of patients, respectively; 22% were RAS/BRAF wild type and 6% had unknown mutational status. 17% (N=5/29 reported) of patients were identified with Lynch syndrome (germline mutation). Of the surgeries performed in the pre-index period, 76% were for primary tumor resections and 24% for metastatic disease. Most common metastatic sites were hepatic (57%) and peritoneum (50%). Prior to 3L treatment, all patients received combination therapy in 1L with fluoropyrimidine + oxaliplatin (53%) and fluoropyrimidine + oxaliplatin + bevacizumab (11%) being the most common regimens. In 2L, 97% of patients received combination therapy with fluoropyrimidine + irinotecan + bevacizumab (23%), fluoropyrimidine + irinotecan (17%) and fluoropyrimidine + irinotecan + cetuximab (11%) being the most common regimens. For 3L and later, combination therapy was preferred over monotherapy but decreased in usage (75% for 3L vs. 57% for 4L). Fluoropyrimidine + irinotecan with or without an EGFR/VEGF inhibitor was the most common combination regimen (N=12) and 5 patients received regorafenib in 3L. The median OS for dMMR/MSI-H mCRC patients receiving 3L therapy was 9.0 months (95% Confidence Interval (CI): 4.0-14.1). Median OS decreased to 4.1 months (95% CI: 4.0-9.0) when survival data of patients receiving ICIs at 4th or later lines were censored at progression date of prior treatment line. BORR was 5.7% (2 patients with PRs), and 31.4% (11 patients) showed stable disease (SD) for 3L treatment. Real-world clinical outcomes observed for dMMR/MSI-H mCRC patients treated in 3L are suboptimal. Study results suggest a high unmet need that could be addressed with ICIs.

aGvHD is a frequent, and often very serious, complication of allogenic Hematopoietic Stem Cell Transplantation (allo-HSCT). The objective of this study was to describe prophylaxis, clinical presentation, and H&OV among patients developing moderate or severe (grade II-IV, Modified Glucksberg or MAGIC) aGvHD. A retrospective chart audit including adult patients diagnosed with grade II-IV aGvHD following the first allo-HSCT (received between Jan-2016 and Jun-2017), and that had not experienced disease progression before aGvHD, was conducted in Finland and Sweden. Clinical characteristics, aGvHD prophylaxis treatment, H&OV were collected from clinical records since allo-HSCT (index date) until date of data collection, death or loss to follow up. 55 patients were included. Mean (Standard Deviation, SD) age at transplant was 48.3 (14.4) years; 54.5% were male. At transplant, 61.8% of patients were in complete remission and 49.1% presented intermediate disease risk index. Most donors were unrelated (76.4%). Two unrelated and one related donor were HLA mismatched. Peripheral blood accounted for 96.4% of stem cells sources. Myeloablative (81.8%) and fludarabine-based reduced intensity conditioning (76.4%) were the most frequent conditioning types. aGvHD prophylaxis consisted of calcineurin inhibitors (98.2%), methotrexate (96.4%), and in-vivo T-cell depletion (80.0%). 70.9% and 29.1% of enrolled patients developed grade II or III-IV aGvHD. Forty-nine patients (89.1%) were hospitalized, mean (SD) number of hospitalizations was 2.9 (2.7). Each patient spent in hospital a mean (SD) of 48.4 (47.7) days. Reasons for hospital admission were aGvHD (65.3%) and infections (44.9%). Seven patients (14.3%) were admitted to intensive care units. Patients required on average (SD) 11.7 (11.1) outpatients and 0.3 (0.6) emergency visits per year. The present study reports key characteristics, prophylaxis treatment, and H&OV of patients developing moderate and severe aGvHD in Finland and Sweden, showing that aGvHD is associated with considerable healthcare resource utilization in these populations.

In this chapter we enlarge the sampling information considered so far by adding to the data of cost and effectiveness of every patient a set of patient covariates. The covariates indicate certain deterministic physical characteristics of the patient such as age, sex, health status, and semiological variables of the disease. The optimal treatment for the whole patient population is typically suboptimal for subgroups, and hence the adaptation of the cost-effectiveness analysis to this situation is of interest and yields the cost-effectiveness analysis for subgroups. Since the definition of patient subgroups is made in terms of the set of covariates it is important to exclude those covariates that do not have an influence on the disease. This means that for carrying out a cost-effectiveness analysis for subgroups a previous step should be the statistical detection of the influential covariates from the original set of them. A …

CELESTIAL (NCT01908426) is a phase 3 multicenter, randomized, double-blinded, controlled trial of cabozantinib vs placebo in patients with hepatocellular carcinoma who had received prior sorafenib.1 EQ-5D-5L was used to assess health-related quality of life (HRQoL). The objective of this analysis was to derive utility values for cost-effectiveness models. Patients completed EQ-5D-5L at date of first drug dose, every 4 weeks through week 25, then every 8 weeks until 8 weeks post disease progression. UK utility values were obtained using the "crosswalk" developed by Van Hout, et al2. As part of the sensitivity analysis, utility values were also derived using the algorithm based on EQ-5D-5L (Office of Health Economics, 2014). Ordinary least squares, Tobit regression with repeated measurements and mixed model for repeated measurements models were tested including treatment discontinuation, progression status and presence of treatment emergent adverse events (TEAE) grade≥3 as potential predictors. The preferred model was selected based on three criteria: use of repeated measurements, Akaike information criterion (AIC) and residuals. In pre-progression status, 2186 questionnaires were analyzed, with slightly higher mean (SD) utility values in patients still on treatment vs patients who had discontinued treatment, 0.73 (0.21) vs. 0.70 (0.24) (p=0.01). After progression status, 696 questionnaires were analyzed; patients that were still on treatment obtained higher utility values, 0.73 (0.21) vs. 0.69 (0.25) (p=0.02). The multivariable mixed model for repeated measures was selected, including progression status and presence of TEAE grade≥3 as predictors. Treatment discontinuation was discarded due to the high correlation with disease progression. The model estimated an intercept of 0.730, with disutility values associated with presence of TEAE grade≥3 of 0.072 (p<0.01) and disutility values associated with disease progression of 0.030 (p<0.01). In the CELESTIAL study, lower utility values were associated with disease progression and presence of TEAE grade≥3.

Objectives: To analyse the impact of overactive bladder (OAB) symptoms on the work activity of patients in Spain. Method: An observational, multicentre cross-sectional study was conducted with 149 urologists and 131 gynaecologists of Spain and included patients diagnosed with OAB, according to clinical judgment, who were of working age (18-65 years). We collected sociodemographic, clinical and work activity data. The patients filled out the Bladder Control Self-Assessment Questionnaire (B-SAQ) and the Work Productivity and Activity Impairment Questionnaire-General Health (WPAI-GH). The effect of each symptom on the daily and occupational activity was assessed. The results were stratified according to sex and the presence of emergency and urinary incontinence (UI) according to the B-SAQ. Results: We assessed 768 patients (89% women), with a mean (SD) age of 52.5 (9.3) years and 2.6 (2.6) years of OAB progression. The most common symptoms according to the patients were urgent urination (89.8%), nocturia (75.7%), urge incontinence (68.5%) and frequent urination (68.2%). Ninety-six percent of the patients adopted adaptive measures for their OAB. According to the B-SAQ, 543 patients (71%) presented urgent urination; of these, 294 (54%) showed UI. The symptom that most affected work activity was frequent urination (59.8%). According to the WPAI-GH, the patients reported an impact of 32% during the workday (41% in patients with urgent urination and UI) and a toss of work time of 6.5% (9.9% in patients with urgent urination and UI). Conclusions: The symptoms of OAB negatively affect work activity, especially in patients who have urgent urination and UI. (C) 2017 AEU. Published by Elsevier Espana, S.L.U. All rights reserved.

To describe characteristics, treatment patterns and resource utilization in Japanese patients with AUD. Observational, prospective study including patients aged ≥20 years with clinical diagnosis of current AUD (DSM-IV criteria), who were under treatment plan or planned to initiate it during the following 4 weeks. Patients with any physical and/or serious or unstable psychiatric disorder were excluded. Clinical data, resource use and quality of life (QoL) were collected at baseline, 2 weeks and 3 months. 150 patients were enrolled at baseline. 145 and 133 patients returned for visits at 2 weeks and 3 months respectively. Patients had a mean age of 53 years, 82% were males and 61.3% were not living alone. 58.7% patients received outpatient treatment and 41.3% inpatient treatment. The most common therapeutic goal was to abstain completely (89.9%). Non-pharmacological therapy was recommended to all patients and pharmacological therapy to 58.9%, being more common in outpatients (66.3%). At baseline, 33.2% of patients had high/very high drinking risk level (DRL) which decreased to 9.0% at 2 weeks and 8.3% at 3 months. At 3 months, 65.6% of patients with alcohol consumption at baseline presented DRL response (WHO definition). Healthcare professionals most frequently visited during the study period were psychiatrists (66.2%), general practitioners (38.3%) and self-help groups (31.6%). During the study period, 37.6% of patients were hospitalized with a mean duration of 24.1 days and 9.0% of patients were on sick leave. At baseline, patients with low/moderate DRL reported better QoL than patients with high/very high DRL (EQ-5D-5L index: 0.8 vs 0.7; EQ-5D-5L VAS: 68.5 vs 56.8; SF-36 general health: 55.1 vs 39.1). Outpatient treatment was most commonly used to reach alcohol abstinence. A significant number of healthcare resources were used during the study period and patients with high/very high DRL had worse QoL.

ObjetivosConocer el manejo de pacientes varones con síntomas del tracto urinario inferior (STUI) y evaluar el seguimiento de las recomendaciones de las guías de la Asociación Europea de Urología en España.Material y métodosEl estudio MERCURY, epidemiológico y transversal, con 227 unidades de urología participantes en España, evaluó a pacientes varones con STUI mixtos predominantemente de llenado, de los cuales se recogió información sociodemográfica, clínica y de consumo de recursos de los 6 meses anteriores. Adicionalmente, mediante un caso clínico teórico, los investigadores describieron su actitud sobre el manejo de los STUI mixtos con predominio de llenado persistentes tras un tratamiento inicial, en cuanto a pruebas diagnósticas y aproximación terapéutica en la primera y la segunda visita. Las opciones proporcionadas para el manejo de los STUI estaban alineadas con las recomendaciones de la Asociación Europea de Urología.ResultadosSe evaluaron 610 pacientes, de los cuales el 87,7% consumió algún recurso sanitario debido a: visitas al urólogo (79,7%), determinación del PSA (76,6%) y tratamiento con alfabloqueante (37,5%) y alfabloqueante más antimuscarínico (37,2%). En el caso clínico teórico, la actitud del urólogo en la elección de pruebas diagnósticas y tratamiento farmacológico fue principalmente: determinación del PSA (97,7%), tacto rectal (91,4%) y tratamiento con alfabloqueantes en monoterapia (56,6%) en la primera visita; flujometría (48,9%), diario miccional (40,3%) y tratamiento con alfabloqueante más antimuscarínico (70,6%) en la segunda visita.ConclusionesLa actitud de los urólogos en España para el manejo del paciente varón con STUI mixtos predominantemente de llenado se ajusta a las recomendaciones de las guías clínicas europeas.

ObjectivesTo explore the management of lower urinary tract symptoms (LUTS) in men in Spain and assess the compliance with recommendations established in the European Association of Urology (EAU) guidelines.Material and methodsMERCURY was an epidemiological and cross-sectional study which involved 227 Urology Units across Spain assessing adult male patients with mixed LUTS and persisting storage symptoms. Sociodemographic, clinical and resource use data for the 6 months prior to study inclusion were collected. Additionally, through a theoretical clinical case, clinicians described their attitude toward the diagnostic and therapeutic management of males with mixed LUTS and persisting storage symptoms during the first and second visits. Answer options given to clinicians about LUTS management were aligned with those recommended by EAU guidelines.Result610 patients included in the study were evaluated. 87.7% of them consumed some health resource mainly due to: urologist visits (79.7%), PSA determination (76.6%) and treatment with alpha-blockers (37.5%) and alpha-blockers plus antimuscarinics (37.2%). According to the theoretical clinical case, urologists preference toward diagnostic tools and pharmacological treatment in first visit were mainly PSA determination (97.7%), digital rectal examination (91.4%) and treatment with alphablockers as monotherapy (56.6%), whereas in the second visit uroflowmetry (48.9%), voiding diary (40.3%) and treatment with alpha-blockers plus antimuscarinics (70.6%) were mainly preferred.ConclusionsUrologists attitude toward management of male patients with mixed LUTS and persisting storage symptoms is aligned with that recommended in the EAU guidelines.

Abstract Aims: To estimate a preference-based single index for the disease-specific instrument (AcroQoL) by mapping it onto the EQ-5D to assist in future economic evaluations. Materials and methods: A sample of 245 acromegaly patients with AcroQoL and EQ-5D scores was obtained from three previously published European studies. The sample was split into two: one sub-sample to construct the model (algorithm construction sample, n = 184), and the other one to confirm it (validation sample, n = 61). Various multiple regression models including two-part model, tobit model, and generalized additive models were tested and/or evaluated for predictive ability, consistency of estimated coefficients, normality of prediction errors, and simplicity. Results: Across these studies, mean age was 50–60 years and the proportion of males was 36–59%. At overall level the percentage of patients with controlled disease was 37.4%. Mean (SD) scores for AcroQoL Global Score and EQ-5D utility were 62.3 (18.5) and 0.71 (0.28), respectively. The best model for predicting EQ-5D was a generalized regression model that included the Physical Dimension summary score and categories from questions 9 and 14 as independent variables (Adj. R2 = 0.56, with mean absolute error of 0.0128 in the confirmatory sample). Observed and predicted utilities were strongly correlated (Spearman r = 0.73, p < .001) and paired t-Student test revealed non-significant differences between means (p > .05). Estimated utility scores showed a minimum error of ≤10% in 45% of patients; however, error increased in patients with an observed utility score under 0.2. The model’s predictive ability was confirmed in the validation cohort. Limitations and conclusions: A mapping algorithm was developed for mapping of AcroQoL to EQ-5D, using patient level data from three previously published studies, and including validation in the confirmatory sub-sample. Mean (SD) utilities index in this study population was estimated as 0.71 (0.28). Additional research may be needed to test this mapping algorithm in other acromegaly populations.

ObjetivosAnalizar el impacto de los síntomas de vejiga hiperactiva (VH) sobre la actividad laboral de pacientes en España.MétodoEstudio observacional, multicéntrico y transversal con 149 urólogos y 131 ginecólogos de España que incluyeron pacientes diagnosticados de VH según criterio clínico, en edad laboral (18-65 años). Se recogieron datos sociodemográficos, clínicos y de actividad laboral. Los pacientes cumplimentaron el Cuestionario de Autoevaluación del Control de la Vejiga (CACV) y el Work Productivity and Activity Impairment Questionnaire-General Health (WPAI-GH). Se evaluó el impacto de cada síntoma sobre la actividad diaria y laboral. Los resultados se estratificaron según género y presencia de urgencia e incontinencia urinaria (IU) según el CACV.ResultadosSe evaluaron 768 pacientes (89% mujeres), con una media (DE) de edad de 52,5 (9,3) años y 2,6 (2,6) años de evolución de VH. La sintomatología más frecuente según el paciente fue urgencia (89,8%), seguida de nicturia (75,7%), incontinencia urinaria de urgencia (68,5%) y frecuencia (68,2%). El 96% adoptaron medidas adaptativas para la VH. Según el CACV, 543 pacientes (71%) presentaban urgencia y de estos, 294 (54%) presentaban IU. El síntoma que más afectaba la actividad laboral fue la frecuencia (59,8%). Según el WPAI-GH, los pacientes refirieron un impacto del 32% durante la jornada laboral (41% en pacientes con urgencia e IU) y una pérdida de tiempo de trabajo del 6,5% (un 9,9% en pacientes con urgencia e IU).ConclusionesLos síntomas de VH impactan negativamente sobre la actividad laboral, especialmente en pacientes que presentan urgencia e IU.

In the context of gradual implementation of economic evaluation of medicines, the Catalan Health Service (CatSalut) aimed to review decisions made and cost-effectiveness criteria considered by seven Health Technology Assessment (HTA) agencies for a sample of 49 oncologic indications. Included agencies were HAS (France), IQWIG (Germany), NICE (United Kingdom), PBAC (Australia), pCODR (Canada), SMC (Scotland) and TLV (Sweden). Reports available online on agencies' websites covering the target indications until October 2015 were reviewed in order to extract information on decisions made and criteria considered. For those reports including an EE, relationship between the type of recommendation and the estimated incremental cost-effectiveness ratio (ICER) was assessed. Overall, 294 reports were identified. TLV (97%) was the agency with a highest proportion of positive opinions, followed by HAS (96%), IQWIG (83%), pCODR (81%), PBAC (60%), SMC (55%) and NICE (45%). More than half of the recommendations made by SMC, TLV, pCODR and PBAC were linked to population restrictions or specific conditions of use. ICERs reported for indications recommended with no restriction or condition were below 50.000€ per quality adjusted life years (QALY) by NICE, PBAC, pCODR and SMC. Although there is variability on ICERs reported across agencies, there is a positive correlation between the reported ICER's threshold and the decision's restriction. High variability in decisions and reported ICERs was observed across agencies, which could be due to both technical (different decision criteria) and strategic (binding decision and its impact in the use of drugs) reasons.